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Would Washington’s FDA Fix Cure the Patients or the Drug Industry?

A bill that would speed up approval for medications and medical devices shows how a major initiative can get traction even in the midst of Washington gridlock — but critics say all the lobbying is drowning out some warnings about patient safety.

In Congress, however, things are looking better for the manufacturers. Legislation is advancing that would speed up the FDA’s approval process for medications and medical devices, offering a rare example of how major initiatives can get traction even in today’s gridlocked Washington.

The industry has mounted a major lobbying and public relations push for the 21st Century Cures Act. The bill, in turn, has garnered an unusually broad range of support, ranging from Republican lawmakers and conservative think tanks to the White House, patient advocacy groups, Democrats and nonprofit organizations that are typically leery of deregulatory efforts by industry. One reason: Lawmakers softened up the usual opponents of looser rules with a big carrot — billions of dollars in new federal medical research funding for the National Institutes of Health. After years of austerity, that money is awfully difficult to turn down.

But the enthusiasts have left a small band of critics warning that bipartisan consensus does not necessarily affirm the bill’s worth. Far from showing that Washington can still get big things done, they say, it shows how a lobby can blow past skeptics if the pot of resources is sweet enough. They maintain that the bill, which easily passed the House in July and has a counterpart soon to be introduced in the Senate, hasn’t received the scrutiny that such sweeping legislation deserves.

“Expanding NIH funding in a substantive amount is a grand and wonderful thing,” said Susan Wood, a former assistant FDA commissioner for women’s health who is now a professor at George Washington University. “But the price of that expansion should not be the gutting of the FDA.”

Wood’s criticism is echoed by other former FDA officials including David Kessler, who was appointed commissioner by President George H.W. Bush, as well as by two Harvard medical school professors who argued in a leading journal that the bill “could lead to the approval of drugs and devices that are less safe or effective than existing criteria would permit.”

For their part, the bill’s proponents say it would spur innovation, particularly when it comes to finding cures for rare diseases — of the 10,000 or so known diseases, 7,000 are considered rare and treatments exist for only 500. Francis Collins, director of the NIH and a leading champion of the bill, says it now takes “around 14 years and $2 billion or more” to develop a new drug and notes that all but five percent of drugs fail during development.

If the legislation passes the Senate and is signed by President Obama, the FDA would be encouraged to develop faster routes to the approval of new products. Under the current system, most new drugs and devices must pass through multiple levels of clinical trials that can take years to conclude. One alternative would be to make more frequent use of so-called “biomarkers” that gauge physical responses to a drug rather than waiting for the final results from a patient trial.

To increase the incentive for drug makers to seek cures for rare diseases, the bill also would grant an extra period of exclusive marketing rights to a company if an existing drug were approved to treat a rare disease after having been previously approved for a different disease.

“The 21st Century Cures legislation is viewed very positively by both political parties and the public at large,” said Michael Castle, a former House Republican from Delaware who is the vice chairman of Research America, an organization that lobbies for biomedical research funding. “If you get down to a list of substantive legislation that has actually a chance of passage now, something like 21st Century Cures is very high on that list.”

The legislation is responding, in part, to the demand from many patients’ groups for medical breakthroughs. “It doesn’t mean you give the industry free rein, but are you really protecting the public if you’re preventing real cures from getting to them?” said Brian Baird, a former Democratic congressman from Washington state who supports the bill.

The bill’s critics have argued that the FDA has already greatly streamlined its approval processes. A recent analysis by Forbes found that so far this year the FDA has rejected only three never-before marketed drugs, and approved 25, an approval rate of 89 percent, up from 66 percent just seven years ago. “We’re the fastest regulatory agency in the world,” said Gregg Gonsalves, a prominent HIV activist now working as a research scholar at Yale Law School. “Pharma would just be very pleased to do less work for more gain.”

Stephen Ostroff, now the FDA’s interim commissioner, has said the agency initially had concerns about the House bill, but that officials were reassured by revisions. The version that passed would only encourage the agency to use the alternate approval methods, rather than require them. Still, critics note that the FDA would get only $550 million to administer the new approval processes, far less than it says it needs to do so properly.

The legislation has its roots in a longstanding push by conservative groups to liberate drug and device development from red tape. “Now, I don’t want to get your hopes up, but Phase Three, maybe we’ll take out FDA,” said Newt Gingrich during the Republican Revolution of 1994, when he also called the agency the nation’s “leading job killer.” More recently, the deregulatory crusade against the FDA has been led by conservative think tanks such as the Goldwater Institute and Manhattan Institute, which launched its “Project FDA” to reform the agency so that it provides a “more predictable, transparent, and efficient pathway” for new medications and devices.

The cause was taken up in Congress over the last couple years by House Energy and Commerce Committee Chairman Fred Upton, a Michigan Republican who will be giving up his gavel to term limits next year and is, his colleagues say, eager for a major legislative capstone before he leaves. Upton has received major backing from the drug and device industries—in the last election cycle, they contributed about $370,000 to him and his associated political action committee, according to the Center for Responsive Politics, more than all but two other business sectors.

Besides campaign contributions, the industry has invested in lobbying. The Pharmaceutical Research and Manufacturing Association, which represents drug makers, increased its quarterly lobbying from $3.96 million to $5.44 million as Upton prepared to release the legislation early this year. The Advanced Medical Technology Association, which represents device makers, increased its quarterly lobbying spending from $550,000 to $740,000 in the same period. Drug and device makers themselves also increased their lobbying expenditures, the records show.

But the key for the legislation’s proponents has been to earn support beyond Republicans and the industry. Early on, Upton enlisted help in crafting the bill from Rep. Diana DeGette, a Colorado Democrat, for whom the legislation was a chance to make a mark on the Energy and Commerce committee, and Rep. Gene Green, a Texas Democrat whose physician daughter took great interest in aspects of the bill.

Then many other Democrats, including New Jersey’s Frank Pallone, the ranking member on Energy and Commerce, lined up behind the legislation when Republicans in June added nearly $9 billion in new support for the NIH over the next five years. The agency has seen its funding essentially flat-line for more than a decade at about $30 billion per year. Pallone did manage to scale back many of the “marketing exclusivity” provisions.

The NIH money also brought the bill support from patient advocacy organizations, from the American Cancer Society to smaller groups seeking cures for rare diseases, which the bill’s proponents say would benefit particularly from the deregulatory reforms.

“I don’t see this as a pro-industry bill,” said Ellen Sigal, the founder and chairwoman of the Friends of Cancer Research. “It’s a bill for innovation and research at basic levels.” She added: “It’s hard, frankly, not to support it. There are very few people who are not supporting it.”

The promised NIH money also brought on board major universities, which carry out about $15 billion of all NIH-funded research. “It was the investment in NIH that led everyone to get behind it,” said Atul Grover, chief public policy officer at the Association of American Medical Colleges. “As soon as we talked about innovation, people said, look, you can try to grease the skids on the approval process, but if we’re not investing as a nation in research, then this other stuff is not going to make much difference. You have to invest in cures to get them.”

The list of entities lobbying on the bill now runs to about 1,800 quarterly entries in the Senate’s lobbying database, with more than 1,100 lobbyists registered as working on it, which is staggering even by the standards of Washington. And what has been so beneficial for the legislation is that the vast majority of those entities are not companies or trade associations, which are motivated by bottom-line demands, but patient groups and universities, which have a far more neutral sheen.

“Members of Congress who wouldn’t be responsive to pharma’s lobbying did respond to universities’ lobbying or to patients’ lobbying,” said Diana Zuckerman, president of the National Center for Health Research, an advocacy group that has spoken out against the legislation. “It was a perfect storm of lobbying.”

In fact, there is considerable overlap between the sets of advocates. Drug and device makers have long provided financial support to many patient and disease groups—some of the money that those groups are spending in pushing for the legislation is also coming from industry coffers. Similarly, many academic researchers whose institutions are lobbying for the legislation in Washington have received consulting and speaking fees from the industry.

The lobbying has also gotten a big boost from Michael Milken, the former junk-bond king who took up the cause of medical research after surviving prostate cancer. Milken has been hosting events in Lake Tahoe, New York, and elsewhere to bring members of Congress together with researchers, patient advocates and industry executives who support the bill.

Meanwhile, an array of the bill’s promoters, including industry representatives, patient groups and scientific associations, are monitoring the legislation’s progress in weekly conference calls and monthly meetings at the office of the American Association of Medical Colleges, regular contacts that the association says it has been hosting for years to push for funding.

The overlap was on display recently at the annual luncheon for one of the nonprofit groups backing the bill, Research America. At the Newseum in Washington, dozens of industry officials, patient advocates and academic researchers mingled with the event’s sponsors, which included the drug companies Astellas, Shire, Janssen, Celgene and Gilead, as well as AdvaMed, the device lobby. Various panel discussions ranged widely across the challenges facing medical research, but throughout the event there was a steady drumbeat urging those in attendance to keep pushing Congress to pass 21st Century Cures.

“We want them to hear us in the Capitol,” said Research America director Mary Woolley as she kicked off the event. “Decisions made just a few blocks from here this fall will be consequential.” (Research America itself receives support from the industry to help cover its costs, which includes Woolley’s roughly $500,000 in annual compensation.)

She was followed by Jeffrey Bloss, Astellas’ senior vice president for medical affairs, who hailed the “groundswell of support” and “massive effort” for the bill. “We need to count on your impassioned advocacy for these changes,” he said.

In an interview afterward, Woolley hailed the coalition behind the legislation. “It’s a very broad consensus—as broad as you can have,” she said. “It’s patient groups, it’s physicians, it’s industry, it’s the academic community, it’s everybody. It’s a goal America can embrace. This is legislation that can make everybody look good.”

And she dismissed the notion that concerns about the bill’s impact on drug safety were being cast aside. “Industry are people too, and they’re patients too,” she said. “The idea that industry is just in this to peddle toxic drugs to sick people is absurd.” She added, “You’d have to think that people from industry and their families are exempt from disease. It’s preposterous. Mistakes hurt everybody.”

Also buttressing the coalition are experts and organizations that in the past have sometimes cautioned against FDA deregulation. One of the bill’s strongest early proponents was the Bipartisan Policy Center, which in January announced a one-year initiative to overhaul the FDA led by former Senate majority leader Bill Frist, the Tennessee Republican and physician.

The bill has also gotten vocal backing from the center-left Brookings Institution, whose director of health care policy, Mark McClellan, served as FDA commissioner under George W. Bush. “None of [the bill’s reforms] is replacing or modifying the FDA’s standards that it needs to be confident that a drug is safe before approval,” he said. “It’s just modifying the evidence that can be brought to bear in making that decision.” McClellan said his support for the bill had zero connection to the funding Brookings receives from drug and device companies, which includes between $500,000 and $999,000 from Genentech and between $100,000 and $249,000 from Amgen. “Those are gifts to the overall institution and they are a small fraction of support to the institution,” he said.

The bill has even gotten support from Pew Charitable Trusts, which has in the past taken the lead in advocating for drug safety. More recently, though, it has taken up the cause of developing antibiotics to combat dangerous infections, and the legislation includes language to speed the development of new antibiotics. Allan Coukell, Pew’s director of health programs, says the organization has endorsed only that portion of the bill, along with a section on prescription painkiller abuse. “I have to limit myself to talking about the provisions we’re working on,” he said. But in promoting those sections of the bill, Pew has also not issued public criticisms of other sections of the bill, to the dismay of some of its usual allies.

“The most difficult thing for the consumer-protection groups has been seeing these seemingly nonpartisan groups sweeping in and embracing the bill as if it’s good for science when everything shows it has the opposite effect,” said Vijay Das, a health care advocate with the watchdog group Public Citizen.

The legislation passed the House by a lopsided 344—77 vote in July. All eyes are now on Lamar Alexander, the Tennessee Republican who chairs the Senate Health, Education, Labor and Pensions Committee, which is expected to release its own version of the bill soon. Alexander has also spoken out for the need to speed up drug approvals—he co-authored a Bipartisan Policy Center report calling for an FDA overhaul—which leaves it likely that the deregulatory language in the Senate version will mirror that in the House. What remains to be seen is whether the Senate will match the House in mandating additional billions for the NIH, an approach that would break from the Senate’s traditional appropriations process.

But David Ross, a former deputy director of drug evaluation at the FDA who now oversees HIV, hepatitis and public health pathogen treatment for the Veterans Administration, still questioned the bill’s underlying justification — that it would result in a higher number of effective drugs getting to market much faster.

“We definitely need more effective drugs, but just calling something effective doesn’t make it so,” Ross said. “It’s a little like gluing some feathers together and calling it a duck. Most drugs that go into studies don’t make it not because the FDA is too strict, but because they don’t just work.”

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